Q4 2024 Syndax Pharmaceuticals Inc Earnings Call
Kip Meintzer, Nadav Zafrir, Kip Meintzer, Roei Golan
Kip Meintzer, Nadav Zafrir, Kip Meintzer, Roei Golan
Sharon Clary: Good day, everyone, and welcome to the Syndax fourth quarter and full year 2024 earnings conference call. Today's call is being recorded. If you'd like to ask a question following the company's prepared remarks, please press star five during the call. At this time, I'd like to turn the call over to Sharon Clary, head of investor relations at Syndax Pharmaceuticals.
Sharon Clary: Before we begin I'd like to remind you that any statements made during the call that are not historical are considered to be forward looking statements within the meaning of the private Securities Litigation Reform Act of 1995.
Sharon Clary: Actual results may differ materially from those indicated by these statements as a result of various important factors.
Sharon Clary: Getting those discussed in the risk factors section in the company's most recent report.
Sharon Clary: Our Form 10-K as well as other reports filed with the SEC.
Sharon Clary: Any forward looking statements made represent our views as of today March three 2025 only.
Sharon Clary: A replay of this call will be available on the company's website www dot syntax dotcom. Following its completion, what's that I am pleased to turn the call over to Michael Metzger, Chief Executive Officer of Synthetics.
Michael Metzger: Thank you Sharon good morning, everyone and thank you for joining us on the call today, starting with slide three we made exceptional progress in 2024 with the FDA approval of two first in class medicines that address major unmet needs a truly remarkable achievement, which puts index and the <unk> category as a biopharmaceutical company.
Michael Metzger: Today marks another major milestone in the company's evolution as we report our first quarter with product revenue.
Michael Metzger: As a reminder, on November 15th 2024, the FDA approved revenue forge our first in class <unk> inhibitor for relapsed refractory acute leukemia patients with eight Kmt <unk> translocation. Just five days later, we had product in the channel with immediate ordering a very fast timeline that highlights the caliber of the team there.
Michael Metzger: We have built to support our transition into a leading commercial oncology company.
Michael Metzger: We are off to a strong start with the U S launch of <unk> and are very encouraged by the patient the early patient demand breadth of prescribing and coverage from payers.
Michael Metzger: For the fourth quarter of 2024, we reported $7 7 million and <unk> net revenue from the initial five weeks of the launch. These early results reflect the high unmet need and demand that <unk> addresses and the strong execution of our organization to deliver the product.
Michael Metzger: We are very excited about the opportunity we have with <unk>, especially given the clinical data supporting the significant opportunity for future label expansions. We recently reported the first positive pivotal dataset in relapsed or refractory mutant NPM, one AML and have continued to report data that show strong evidence of synergistic.
Michael Metzger: <unk> in combination with standard of care therapies across newly diagnosed and relapsed or refractory patients with <unk> rearrangement, the <unk> mutations or <unk> 98 rearrangements.
Michael Metzger: We are preparing to file a supplemental new drug application or NDA next quarter that could expand the indicate indicated population for <unk> mutant <unk> AML around the end of the year.
Michael Metzger: In parallel we are also aggressively advancing our clinical development strategy in the frontline setting to support label expansions and inclusion in the NCC and guidelines. We are very confident that we are well positioned to lead in the frontline setting building off our leadership in the space as the first and only company with approved <unk> inhibitor.
Michael Metzger: Shifting gears to our second product Nick timber.
We are thrilled to have launched Nick Tim, though in chronic graft versus host disease or C. Gvhd in late January in partnership with insight the leader.
Michael Metzger: Graft versus host disease.
Michael Metzger: Tivo is the first and only FDA approved treatment for chronic gvhd that targets CSF, one or to reduce the drivers of inflammation and fibrosis.
Michael Metzger: Like revenue forged Nick timber has significant potential for expansion to unlock those opportunities we and insight are advancing multiple trials. Two trials are underway that are evaluating <unk> in combination with standard of care therapies in newly diagnosed chronic gvhd patients, including a phase II trial with Jakafi and <unk> phase III.
Michael Metzger: Trial with steroids.
Michael Metzger: Enrollment is also ongoing for Mac spire, our phase II trial in idiopathic pulmonary fibrosis or IPF with topline data expected in 2026th.
Michael Metzger: Turning to our financial position in November we further strengthened our balance sheet through a deal with royalty pharma the Premier company with the largest by the end of <unk>.
Michael Metzger: Large buyer of biopharmaceutical royalties, we received $350 million upfront in exchange for a capped royalty on U S. Net sales of Nick timber deal that highlights an active those multibillion dollar potential.
Michael Metzger: We are in a strong financial position with a balance sheet that is expected to fund our operations through profitability.
Michael Metzger: I'll now turn the call over to Steve to discuss our commercial progress in more detail Steve.
Steve: Thank you Michael it's really a pleasure to be on the call today to provide an update on the launch of <unk> and the Kimbo starting with slide four it has been incredibly rewarding for me and our entire team to be a part of bringing <unk> to patients. We are very encouraged by the progress. We have made in the early phase of the launch as Michael stated in the initial <unk>.
Steve: Five weeks of launch we generated $7 7 million and net revenue.
Steve: We estimate that approximately one third of the net revenue for this first partial quarter represents inventory at our specialty pharmacies and specialty distributors and the remainder represents patient demands patient demand was driven primarily by new patient starts with only a small number of patients transitioning from our expanded access program or <unk>.
Steve: To paid drug.
Steve: All of the pre and post launch work, we have done with Hcp's with payers and other stakeholders is truly paying off we are seeing high product awareness among clinicians and the feedback from our customers has been overwhelmingly positive regarding the product profile and the overall ease of access to the medicine, which really speaks to the efficiency of the limited distribution.
Steve: <unk> model that we have established our distribution model includes partnerships with two of the leading specialty pharmacies for oncology products as well as the network of specialty distributors and our own dedicated patient support program known as indexes.
Steve: Thanks to the systems, we have in place the high quality of our internal team and the work we've done with payers. We are seeing patients get approved for coverage quickly and a rapid time to first fill which is the time it takes from initiating a <unk> prescription to the patient actually starting medication.
Steve: Turning to slide five.
Steve: As Q4 was a partial quarter of sales I'd like to provide some high level color on the trends we're seeing in the first quarter of 2025 that provides additional insight into the health of the launch.
Starting with the top of the slide we are seeing very encouraging early breadth and depth of revenue forged prescribing as of the end of February 33% of our high priority or tier one and tier two accounts have ordered <unk>. These.
Steve: These accounts are the centers of excellence in the medium to large academic institutions that represent two thirds of the patient opportunity.
Steve: While we prioritize our efforts at these top accounts. We're also calling on the entire universe of 2000 relevant accounts across the U S and are seeing orders from accounts beyond our tier one and tier two accounts, including from community practices, which reflects the high unmet need and the ease of use of revenue forge, notably the majority of accounts.
Steve: I've ordered have written more than one prescription for <unk>.
Steve: We're leveraging data from multiple sources to help our field teams identify and engage providers at the time when they may have an appropriate patient in their care, which is proving to be an effective approach.
Steve: We're seeing a mix of patients prescribed <unk>, which is consistent with our broad label, which includes both adults and pediatrics as well as patients with any lineage of relapsed or refractory acute leukemia with cancer to a translocation, including AML.
Steve: L or M P a L.
As expected revenue for just being prescribed to patients across the treatment continuum, including patients on their first relapse as well as patients with more advanced disease based on feedback from physicians the dire need in this patient population and the lack of any other targeted therapies for these patients we expect to see red before it has rapidly become the standard.
Steve: Of care for patients with <unk> translocations at the time of their first relapse.
Steve: Turning to the payer front and market access.
Steve: We did extensive work educating payers before and after launch and we're benefiting from these efforts as well as the rapid inclusion of <unk> and the NCC guidelines for AML in a L. L.
Steve: Payers recognize the urgent unmet need and the clinical value revenue forge and we're seeing reimbursement in all payer channels, including commercial Medicare part D as well as Medicaid.
Steve: Roughly three months into the launch and we're seeing very strong formulary uptake with formal coverage policies in place for approximately 56% of commercially covered lives and 53% of all managed care lives, which includes all commercially covered plus Medicare and Medicaid lives.
Steve: Importantly, we are pleased to report that the vast majority of prescriptions are being reimbursed by payers. Despite formulary coverage still building.
Steve: As anticipated revenue for just being approved through the medical exception process on formulary coverage is not yet in place.
Steve: Day in and day out our team and specialty pharmacy partners are moving mountains when necessary to ensure that patients get the treatment they need with the speed and care, we would all want to see if it was our loved one in need.
Steve: This commitment to delivering a best in class customer experience is the right thing to do for patients and that delivering on this very commitment will be one important factor that drives long term competitive immunity.
Steve: Turning to slide six.
Steve: <unk> is well positioned for near term and long term growth and success in.
Steve: In the near term our current indication provides us with the opportunity to target an estimated 2000 patients in the U S with relapsed or refractory acute leukemia with <unk> translocation.
Steve: When you take the price of RMB for Egencia account and an estimated average treatment duration of about nine months across various patient experiences. This initial opportunity represents a $750 million market opportunity and.
Steve: And with positive pivotal data in relapsed or refractory mutant NPM, one AML, we have a near term opportunity to receive a second indication that could expand our target population in the U S to 5000 to 6500, <unk> and NPM, one patients, which together represent a $2 billion market opportunity.
Steve: <unk> is the first and only FDA approved <unk> inhibitor on the market today and our teams are working with urgency to capitalize on the advantages of being first to market.
Steve: We know that once physicians become familiar with the new therapy. They tell us the bar is high to switch to another product with the same mechanism.
Steve: <unk> is also the only <unk> inhibitor with positive pivotal data in both relapsed or refractory can't do too to a rearranged acute leukemia and mutant <unk> AML and in both adult and pediatric patients the consistency and breadth of the data supporting <unk> as a strong strategic advantage because physicians prefer to have one effective <unk>.
Steve: Safe drug that they can prescribe across a wide range of patients.
Steve: Looking ahead, we are well positioned to extend our leadership in the relapsed refractory setting into the frontline setting and we have a robust development plan underway that is designed to unlock the total market opportunity that exceeds $4 billion in the U S across the relapsed refractory and frontline settings.
Steve: Moving to slide seven.
Steve: In mid January we and our commercialization partner insight announced that the FDA approved Nick timber and nine milligram in 'twenty two milligram vial sizes in late January we launched the product and received our first orders and in mid February we had the opportunity to drive awareness of niche timber tandem largest conference for our clinicians who perform stem cell transplants.
Steve: While it is still very early in the launch we are encouraged by the level of interest in the timber which speaks of the high unmet need and the products compelling clinical profile.
Steve: Together with insight we are working to bring this new options to the approximately 6500 chronic gvhd patients in the U S who require three or more lines of therapy.
Steve: Addressing the needs of this patient population represents an attractive commercial opportunity for example in the three years since the launch of <unk> most of the reservoir rock in other drug indicated for the same line of treatment as Nick timber.
Steve: Net sales continue to grow in the high double digits and suggests that the drug is now annualized at over $500 million in U S sales.
Steve: With a new mechanism of action and strong clinical data showing remarkable responses in heavily pre treated patients. We believe that Nick timber, we will be able to capture a significant portion of the one $5 billion to $2 billion estimated total addressable market for third line or later chronic gvhd treatment in the U S.
Steve: We are confident that we have just started to scratch the surface of the opportunity with Nick Tivo and are thrilled to be working with insight on trials designed to move the product into earlier lines of treatment for chronic gvhd and other inflammatory and fibrotic diseases, starting with IPF.
Steve: With that I'll hand, the call over to Neal to discuss the work we are doing to develop revenue minimum for the treatment of acute leukemias across the treatment continuum. Thanks, Steve.
I'm pleased to provide an update on the rough amount of development programs starting on slide eight with a near term opportunity we have to expand into relapsed or refractory mute to MTN one ml.
Speaker Change: Given the high unmet need and lack of approved therapies that target. This population. We are pleased to have reported the first positive pivotal data set.
Speaker Change: In November 2024, we announced that the primary endpoint was met in the protocol defined efficacy population of 64 adults with relapsed or refractory B NPM, one AML in the phase II cohort of the augment 101 trial.
Speaker Change: Heavily pretreated population, including 75% with prior venetic tax exposure nearly half of patients achieved a response in 23% achieved CR CRH.
Speaker Change: Consistent with other datasets that responses were deep was 64% of CRC rates responders, achieving <unk> negativity.
Speaker Change: <unk>, 17% of responders proceeded to stem cell transplant, which is a meaningful outcome in a population that is older and less fit for transplant them for example patients with <unk> arrangements.
Overall, the data are compelling and we are confident that they could support regulatory approval further solidifying our leadership position.
Speaker Change: On the positive pivotal data we reported results in December 2024 from a larger post hoc analysis, which included additional mutant <unk> patients from the phase II cohort, who met the efficacy evaluable criteria.
Speaker Change: These results were highly consistent with those for the primary analysis with an overall response rate of 48% CR Cri rate of 26% amongst 77 efficacy evaluable patients.
Speaker Change: Looking ahead, we expect to file the NDA in the second quarter and believe we could receive approval around year end.
Speaker Change: Also in the second quarter, we expect to publish the pivotal NPM one data and believe this could serve as the basis for the expansion of the listing for <unk> and the <unk> guidelines for AML ahead of the potential FDA approval in this population.
Speaker Change: Turning to slide nine we are advancing revenue amounted for the treatment of genetically defined acute leukemias across the treatment continuum and are well positioned to establish it as the preferred menin inhibitor for both relapsed refractory and newly diagnosed patients in combination with the standards of care.
And the frontline, we're advancing a thoughtful combination of syntax sponsored and investigator initiated trials of <unk> in combination with standard of care in patients ineligible are unfit for intensive chemotherapy as well as those who are eligible are fit for intensive chemotherapy.
Speaker Change: Our strategy prioritizes addressing population with the greatest unmet needs speed to data on the generation of a regular cadence of potentially practice changing evidence that could support label expansion.
Speaker Change: And or infusion of MCC and guidelines.
Speaker Change: Moving to slide 10.
Speaker Change: Our next steps in the frontline and collaborations with the Hogan network, we are initiating a pivotal randomized placebo controlled trial of <unk> with <unk>.
Speaker Change: In newly diagnosed patients with <unk> mutations or came to <unk> rearrangements, who are ineligible for intensive chemotherapy either due to their age or comorbidities.
Speaker Change: Our partnership with a well established over our network provides significant advantages for trial.
Speaker Change: Efficiency by allowing us to quickly enrolled patients.
Speaker Change: Globally, while conducting the trial with the highest quality.
Speaker Change: The pivotal trial will build on the encouraging data from beat AML at Phase one trial evaluating <unk> in newly diagnosed <unk> rearrange to AML patients who are unfit for intensive chemotherapy.
Speaker Change: Latest data show that the overall response rate was 100% of the mrna negative rate was 95% among 37 efficacy evaluable patients.
Speaker Change: These results highlight the potential for a heavy amount of to enhance the responses observed with <unk> in this population.
Speaker Change: Data also demonstrate that revenue amount of can be combined with standards of care in the frontline setting.
Speaker Change: Turning to the frontline population that is eligible to receive intensive chemotherapy. We expect to report data in the second half of 2025 from an ongoing phase one trial of revenue amount up with seven plus three or intensive chemotherapy.
Speaker Change: What's the plan to initiate multiple trials of revenue management in combination with standards of care in newly diagnosed AML patients who are eligible to receive intensive chemo starting in the second half of 2025.
We are confident that we are poised to lead and win in the frontline setting and we recognize that speed to data will be key.
Speaker Change: Furthermore, we will be in a position to avail ourselves of any opportunities that may emerge to accelerate timelines with the use of alternative endpoints.
Speaker Change: For example, as leaders in the managed space, we will continue to partner with the foundation for the National Institutes of Health Biomarkers consortium on MLD in AML to establish <unk> as a validated biomarker in the disease.
Speaker Change: With that I'll hand, the call over to Keith to discuss our financials.
Keith: Thank you Neil.
Speaker Change: Earlier. This morning, we reported fourth quarter and full year 2024 financial results and press release.
Speaker Change: For today's call I'll touch on a few highlights from our fourth quarter on slide 11.
Speaker Change: We're pleased to report seven $7 million of revenue <unk> net revenue in the fourth quarter of 2024.
Speaker Change: Operating expenses in the fourth quarter were 104.0 million.
Speaker Change: And included $65 $5 million of research and development expense and.
Speaker Change: And $37 $7 million of selling general and administrative expense.
Speaker Change: Turning to the balance sheet.
Speaker Change: We continue to maintain a strong financial position with $692 $4 million of cash equivalents and short and long term investments as of the end of the year.
Speaker Change: We continue to expect that our cash together with our anticipated product revenue and interest income will enable the company to reach profitability.
Speaker Change: For the first quarter of 2025.
Speaker Change: The company expects R&D expenses to be $65 million to $70 million.
Speaker Change: In total R&D, plus SG&A expenses to be $105 million to $110 million.
Speaker Change: For the full year of 2025, the company expects R&D expenses to be $260 million to $280 million.
Speaker Change: And total R&D, plus SG&A expenses to be $415 million to $435 million, which includes an estimated $45 million and noncash stock compensation expense.
Speaker Change: The company is not providing revenue guidance at this time.
Speaker Change: Ahead of reporting the first sales predictable with our first quarter 2025 results I would like to take a moment to briefly review how we will recognize revenue for that partnered products.
Speaker Change: Slide 12 provides an illustrative example of accounting for yourselves and Nintendo and is not intended to provide any margin or other guidance.
Speaker Change: We will record 50% of the commercial profit.
Speaker Change: <unk> net product revenue minus the cost of sales and commercial expenses.
Speaker Change: During a period, where there's net commercial profit predictable is in the top examples.
Speaker Change: Our 50% share of the net profit will be recognized in our P&L as collaborative arrangements revenue.
Speaker Change: Of note the expenses that are deducted from the topline to timber revenue to arrive at the collaborative revenue will not include the synthetic royalty to royalty pharma.
Speaker Change: The upfront $350 million that we received from royalty pharma as liability classified on the balance sheet and the $13, 8% royalty payments will be reflected on our financials as a combination of interest expense and amortization of the liability principal amount.
Speaker Change: During a period, where there was a net commercial loss for Nick Tivo is in the bottom of example on the slide.
Speaker Change: Our 50% share of the net commercial off loss would be included in operating expenses designated as a separate line called share of collaboration loss.
Speaker Change: Any milestone revenue from various commercial and regulatory milestones that we received from insight will be recorded as milestone revenue on our income statement.
Speaker Change: As a reminder, research and development expenses, including regulatory and CMC expenses are shared 50 545 in the U S and our 45% share is included in the income statement as part of our R&D expense.
Speaker Change: Outside of the United States, Instead is responsible for 100% of the development and regulatory expenses.
Speaker Change: We are entitled to receive milestones plus a double digit royalty on ex U S sales.
Michael Metzger: With that let me now turn the call back over to Michael.
Michael Metzger: Yes. Thank you Keith has been a very successful start to the year for <unk> and we are poised for another exciting period as we advance the launches of <unk> and work towards achieving the multiple upcoming milestones that you can see on slide 13.
We are confident that index is well positioned for long term growth. We are excited about the de risked and very significant market opportunities. We have in front of us with the first two differentiated medicines to emerge from our pipeline we.
Michael Metzger: We are laser focused on unlocking the full value of both products and are set up for success with a strong balance sheet and a highly experienced team that has done an exceptional job executing to plan through out the development approval and commercialization of our first two medicines.
Michael Metzger: In closing I want to thank everyone, who has made it possible for US index to bring two novel medicines across the finish line in 2024, I, especially want to thank the patients investigators and study sites, who participated in our clinical trials as well as our dedicated and talented index team members I'd also like to thank our committed long term investors who.
Michael Metzger: To share in our vision and support our work in building <unk> into a leading oncology company and objective that we are well on our way to achieving and with that I'd like to open the call for questions operator.
At this time I'd like to remind everyone in order to ask a question Press Star then the number five on your telephone keypad, if you'd like to withdraw your question Press Star then the number five once again.
Michael Metzger: Pause for a moment to a pilot.
Michael Metzger: Q&A roster.
Speaker Change: The first question will come from Andrew Palm Rama with Jpmorgan. Please go ahead.
Speaker Change: Hey, guys. Thanks, so much for taking my question and congrats on the launch progress here in the early innings.
Speaker Change: Just a quick one for me.
Speaker Change: I know in the press release, you talked about about a third of the <unk> sales were inventory just wondering how we should be thinking about inventory levels throughout 2025, and then I know that you said.
Speaker Change: You're about a third penetrated into your tier one two accounts.
Speaker Change: Yes.
Have you any trends on repeat prescribers from some of the docs given the aggressive nature of Kmt <unk>. Thanks, so much.
Speaker Change: And Bob Thanks for the question. So two questions. So first first question I'm going to direct to Keith which is related to inventory levels. Good morning, Paul.
Good question. Thank you I think our inventory levels as you think forward through the through 2025.
Speaker Change: Probably be thinking about it similar to other specialty launches of rare disease launches.
Speaker Change: As a result of our limited distribution channel, which Steve talked about we're not expecting to see more than two to three weeks of inventory in the channel.
Speaker Change: As we mentioned in our prepared remarks that we saw about approximately one third.
Speaker Change: Stocking.
Speaker Change: And five weeks, we're kind of right there in the sweet spot. So we wouldn't expect the inventory level to change in terms of weeks, but as revenue grows obviously that inventory level on an absolute basis could be expected to grow.
Speaker Change: Great and Steve you want to take the penetration into accounts question. Thanks, John upon the comment we're pretty pleased with where we are we've got 33% as you mentioned of tier one or two accounts. Our biggest accounts account for two thirds of the opportunity that are getting on board. We are confident will will go along the lift and get others to REIT as well in terms of.
Speaker Change: Repeat prescribers something we're looking at carefully and we're not going to provide.
Speaker Change: <unk>, yet or estimates on that but I will say the majority of accounts have have used more than once and thats. Good whether that's a patient getting a refill or finding new patients. I mean this is all about building a habit and the better experience. They have upfront the more likely they are to use the product moving forward and we feel really good about where we are.
Speaker Change: Yeah.
Speaker Change: Thanks, so much guys and congrats.
Speaker Change: Thanks Ana pump.
Speaker Change: The next question will come from the line of Brian <unk> with Stifel. Your line is now open.
Brian <unk>: Good morning, and nice first commercial quarter, two parter for me.
Speaker Change: 66% of tier one two accounts that have not yet ordered what do you expect for the cadence and extent of activation over the next 10 months and then second now with a few months of commercial experience under their belt.
Speaker Change: Do you see revenue forged tracking with some of the historic AML targeted therapy launches. Thank you.
Speaker Change: Okay. Thanks, Brad I appreciate the question so first of all.
Speaker Change: Direct to Steve.
Speaker Change: Yes, so thanks, Brad for the question and I liked the hikes that you think I mean, I think for US. It's a good start to have a little over a third of accounts activate really early and it's a good mix of accounts. It's not just the biggest it's not it's not the small since it's a broad range. We think it represents a ton of opportunity moving forward.
Speaker Change: Not going to comment on on the cadence I think what we've what we what we are doing is activating across all 2000 treatment centers, we're going to focus on the top 200, we think it's just a matter of time for those institutions to get experience. We've got a sales team that is perhaps the most experienced and talented in the industry they've got great relationships, we still.
Speaker Change: I have a ton of momentum.
Speaker Change: So we expect over the course of the year one by one.
Speaker Change: We'll pick each one off as you can imagine the 33% is a blend of tier one and tier two there is far more tier one accounts that have activated.
Speaker Change: And fewer of the tier two accounts, but over time with the right type of effort. They will fall there is unmet need product is <unk>.
Speaker Change: Responding well.
Speaker Change: So we're pretty positive over the course of the coming months.
Speaker Change: Okay, then maybe the second question relative to analyst.
Speaker Change: A analogs I think we've got a unique setup I mean, I think unlike other targeted therapies in the past you've got testing.
For a product like rugby forged identifying <unk> patients, which you may not have had as readily speaking to other targeted therapies. So urgent unmet need is high patient pay.
Speaker Change: Patient diagnostic testing is very high as mentioned the product is very well received by by clinicians and on the payer front things are in a great place that all companies look to get paid claims early on in the launch there is a sense of kind of giving drug away. That's that's not what we're doing we're developing a habit for physicians to understand the cut.
Speaker Change: Our experience they can expect which we believe is a good one so too early to say, where we are relative to those analogs, but feel great about where we are just one partial quarter and in a couple of months and to the start of 2025.
Speaker Change: Great. Thank you.
Brad: Thanks, Brad.
Speaker Change: The next question will come from Peter Lawson with Barclays. Your line is now open.
Peter Lawson: Great. Thanks, so much thanks for taking my questions.
Thanks for the updates.
Peter Lawson: I guess first thing around revenue for us like how that's being used if youre seeing off label use how much for NPM, one if you've seen it being used in combination.
Peter Lawson: And then just regarding the expanded access program just maybe a clarification for me you kind of talked about a rapid transition.
Peter Lawson: You'd mentioned less than 10 patients transitioned.
Peter Lawson: How many patients and less than that.
Peter Lawson: Extended access program.
Peter Lawson: Yes.
Peter Lawson: Yeah. Thanks, Peter maybe Steve again for you off label use first question, Yes. Good question Peter.
Obviously, it's not something we promote against we're only going to promote against the primary indication as you would expect in terms of off label use what we can say at this point. It's anecdotal we know we're getting us some use in MTM won some Houston and combination treatments certainly earlier line types of treatment.
Peter Lawson: Anecdotal overtime, we'll be able to better quantify that but at this early stage. It's it's it's a challenge to do that and in terms of the EAP question as pointed out it's.
Peter Lawson: We're talking EAP transitions were in the single digits all patients that were eligible to be transferred have been.
Peter Lawson: And the AP remains likely use for off label indications and oral solution. That's also the widely used there but.
Peter Lawson: No no additional patients to transfer from AAP at this time.
Peter Lawson: Just maybe a final question has momentum going.
Peter Lawson: First.
Peter Lawson: 25.
Peter Lawson: The momentum is good I mean, I think if you remember we started this back in the fall initially with the M. P. M. One data release, followed by approval followed by Ash believe it or not institutions are still doing their ash reviews, which is ER physicians that didn't make it to the conference. They see the data. So we'll certainly see the data on revenue for us as well as in the <unk> space. So that momentum that we that we saw.
Peter Lawson: <unk> continues to this day.
Peter Lawson: Very high excitement level.
Peter Lawson: Our field team as mentioned is closely connected to the customer community. So opened welcome arms.
Peter Lawson: Physician see unmet need they like what they see in the drug. So I think we'll ride this wave really probably throughout the rest of the year.
Yes, and maybe I would add that the payer coverage seems to be building really very nicely and I think that's.
Peter Lawson: Complement to all of the all the hard work that the team is doing to get it.
Peter Lawson: Get it through the payer systems.
Peter Lawson: Great. Thanks, so much.
Peter Lawson: Thank you Peter.
Speaker Change: The next question will come from the line of Chris <unk> with Goldman Sachs. Your line is now open.
Chris: Great. Thank you very much congratulations on great progress with the initiatives part of the commercial launch many questions have been asked there, perhaps if I could ask a little bit more of an intermediate and longer term strategic question certainly the company has gotten to this point finding assets developing them successfully now commercializing.
Chris: What are your thoughts on what's next what does this company look like in 2030, many arguments foreseeing that if youre, making these investments and a fully integrated company that will be soon turning to what else is on the pipeline based upon your experience capabilities and now commercial presence do you have a vision to that are you.
Chris: Sure that if not might not when might you and are you and the board of mind. Thank you.
Speaker Change: Well Chris.
Speaker Change: So big question here. So thank you for asking it I think the vision for the company as ambitious I think we have done wonderful things to get to this point and not only in licensing and developing two novel medicines, but bringing them to patients and I think we'll do quite well commercially I think.
Speaker Change: The broader vision is to extend these products into earlier line indications and really build on our first mover advantage. So I think that's a longer term vision for these particular products and I think the the extension to additional products, bringing in additional products targa.
<unk> targeted oncology has been our focus I think we will continue to build in oncology and build the pipeline Ulf.
Speaker Change: Ultimately to be able to leverage the organization that we've developed over time. So I think we have a.
Speaker Change: An ambitious vision to have a.
Larger pipeline and to build a company over over several years by 2030, we expect to be.
Speaker Change: Extremely.
Speaker Change: Advancing rapidly advancing company with multiple commercial products in in our in our bag so to speak and have and have the opportunity to continue to get drugs approved.
Speaker Change: The vision long term vision as a standalone company will be focused in the U S and focus in oncology.
And look to partner.
Speaker Change: External in ex U S. As we built the business, but I would say I think we would hope to be a a formidable specialty oncology company by 2030.
Speaker Change: And yes, the board the board and management are quite aligned in this approach.
Perfect. Thank you for the insights.
Kurt: Thank you Kurt.
Speaker Change: The next question will come from the line of Kelly <unk> with Jefferies. Your line is now open.
Kelly: Congrats on a great quarter and thank you for taking my questions.
Speaker Change: Although interest you at any day, but at Shire for patients bearing commutation such as at <unk>.
Kelly: The sequence.
Kelly: Eric.
Kelly: And what would be the reason that I think is entirely at higher rents.
Kelly: Sorry.
And also curious 60 found could you share about the physician feedback on their bill or excuse me. Thank you very much.
Speaker Change: Sure Kelly. Thanks for the question. So first one related to the prioritization why would someone why would a physician use of menin inhibitor versus some of the other targeted therapies I'll direct that to Neil.
Neil: Yeah. Thanks for the question so.
Speaker Change: The answer is in fact, two parts right. So it depends on whether or not the patient is.
Neil: Considered to be eligible or ineligible for intensive chemotherapy.
Neil: The phase III trial that we're currently initiating in the unfit population there is no selection for four.
Neil: Flit three we went into patients as I mentioned in our in the prepared remarks patients with both <unk> and <unk>. There is no approved therapy for patients with <unk> three mutations.
Neil: Who are unfit for intensive chemotherapy.
Neil: And the fit setting at slightly different there are approved therapies and therefore.
Neil: Bill There are approved other approved therapies, then physicians will.
Neil: <unk> four <unk> three inhibitor for patients who have a fifth three fleets.
Neil: With three mutation before before anything else.
Neil: Of course patients can have both flip three in <unk> mutations at the same time and this is a.
Neil: An area of intense interest for us.
Speaker Change: Great. Thank you and then the second question I'll refer to Steve.
Speaker Change: I think the question Kelly was related to physician feedback on the safety profile, so far real world. So, yes, I mean, I think feedback on our whole profile is great from an efficacy standpoint, it's got data that's instructive to us.
Speaker Change: In terms of safety and Tolerability.
Speaker Change: We don't have a black box for the asset that's being managed.
Speaker Change: Qt other aspects of of Tolerability are understood by physicians of labels clear and I'd say the other thing that we do is have a.
Speaker Change: A pretty comprehensive customer facing footprint, we have oncology clinical nurse educators that are in accounts within 48 hours of the drug being prescribed so it's a lot of handholding and helping physicians and their associated staff through through treatment and dosing is clear white age population AML ALLL. So I'd say things are very good there.
Speaker Change: Really nothing getting away of physicians, putting patients on <unk>.
Speaker Change: Thank you.
Speaker Change: Thank you Kelly.
Speaker Change: The next question comes from the line of Michael Schmidt with Guggenheim. Your line is now.
Michael Schmidt: Hey, guys. Good morning, Thanks for taking our questions I had a couple more on the <unk> launch.
Speaker Change: Keith perhaps could you comment on early trends in gross to net adjustment as well as potential.
Michael Schmidt: A free drug program et cetera.
Speaker Change: And then in.
Speaker Change: In terms of early feedback from the market what have you heard about or seen around that.
Speaker Change: Last Monday.
Speaker Change: Yes.
Thanks, so much.
Speaker Change: Thanks, Michael sure. So first first question related to gross to net maybe I'll direct to keep yeah. Good morning, Michael.
Speaker Change: While we're not providing specific numerical figures on gross to net.
Speaker Change: It's tight.
Speaker Change: Think go back to the comments I made earlier about our limited distribution channel.
Speaker Change: That allows us to really keep the gross to net very tight. Additionally, we've not seen nor do we foresee the need to rebate. So for those for those reasons and a few others the gross to net on <unk>.
Has been and we expect it to continue to be very tight.
Speaker Change: And then maybe Steve do you want talk about Michael.
Speaker Change: Michael had asked about free drug program. So once you make a comment on that so we do have a patient assistance program.
Speaker Change: It's used for basically uninsured or underinsured patients I mean, our goal as an organization is to make sure every patient has drug our first priority is to get the claim paid if.
Speaker Change: If the patient can't qualify for paid drug there is that patient assistance opportunity. It's in the single digits. I mean, you often you can see some pressure at launch you could see some pressure as deductibles reset for the beginning of the year I think we've navigated through that incredibly well, it's going to be in the low and the low single digits.
Speaker Change: And then maybe early feedback on the transplant dynamic I think physicians are.
Speaker Change: Obviously very interested in getting patients to transplant certainly for <unk>. We've also seen it for NPM, one as well, but most of these patients are on the younger side for <unk> and so transplant is an objective and we know patients are going to transplant. So they are going to getting drug and then go into transplant.
Speaker Change: And then we expect the vast majority of those patients, especially as theyre treated earlier in their treatment course, or like first relapse I had the opportunity to potentially go back on transplant go back on maintenance after after receiving their transplant in the graph that happened. So I think we're optimistic about how that's unfolding I think it's early days yet.
Speaker Change: Two to really say much more about it but as of today. This is.
Speaker Change: How were feeling very good.
Speaker Change: Thank you.
Speaker Change: Thanks, Michael.
Speaker Change: Our next question comes from the golf in the Delaware with Citi. Your line is now open.
Speaker Change: Michael and team. Thank you and congrats on the first quarter revenues I have three quick ones. If I may 1st by my math. It seems like you have about 125 to 130 patients.
Speaker Change: <unk> net of the inventory, which is about 6% share could you just comment on that.
Speaker Change: If you could be a little more specific regarding how you're defining tier one versus tier two centers and then third for Neil Perhaps you mentioned some of the flip three dynamics I'm. Just curious if you could be a little bit more granular as far as how those phase II trials could look because I could imagine for studies, a <unk> study for those with us.
Speaker Change: Flip three and those without similarly on P. M. One for those within flip three and those without this would be in the state population. So would you have.
Speaker Change: 703, plus that slipped three control arm.
Speaker Change: Those studies respectively. Thank you.
Speaker Change: Great. Good questions Yigal. Thank you. So let me turn the first two Steve talking about the the math that you've that you've done 125 to 130 patients.
Speaker Change: Net of inventory roughly 6% of the population.
Speaker Change: That's your math, so maybe some comments on that yes.
Speaker Change: I appreciate it.
Speaker Change: The math sure.
Speaker Change: And we're just we're not going to provide specifics on patient counts just yet there's really a couple of reasons for what it is just.
Speaker Change: We have visibility to patient level and less than half of our limited distribution channel. It's really just to the specialty pharmacy channel. So we want to be accurate and that will take us some time to do that and I think the second is it. Its just early from a patient perspective I think the second question was around tier one and tier two definition.
Speaker Change: And it's roughly combined about 200 accounts those accounts.
Speaker Change: At present about two thirds of the patient opportunity. So tier one you can think.
Speaker Change: I will say biggest of the big MD Andersen Dana Farber. Those those are typical tier one accounts tier two just tend to be a little smaller it's really just defined by patient opportunity. So these are influential accounts, but may not have the patient volume that the tier ones have two examples there could be like a St jude's or Tampa Gerald just to give you some examples.
Put it in perspective.
Speaker Change: Great and then I guess the third question goes to Neo which is related to the the trials of the fit population specifically around flip retail so thanks.
As you referred to in the prepared remarks.
Speaker Change: It's to initiate multiple trials in the <unk> setting.
Speaker Change: We also alluded to the fact that speak to date is important.
There are of course multiple populations within the fit it's not an imagine its population, including patients with flip three and nine P. M. On cocoa mutations, we're not really talking in detail about our strategy. It's a competitive space, but we are working aggressively to start multiple trials starting out in the second half of this year.
Speaker Change: Okay. Thank you so much.
Speaker Change: Thanks Hugo.
Speaker Change: Our next question comes from the line of David Dye with UBS. Your line is now open.
Speaker Change: Great. Thank you for taking my question and also on a congrats on a great quarter. So two from me as well. So the first one just around the relapse refractory NPM one pivotal data you're planning to submit for <unk> guidelines headquarters here could you maybe talk about the timing of the inclusion of <unk> for <unk>.
Speaker Change: Nancy and guidelines do we think about the inclusion of the FC.
Speaker Change: Before the approval in second half this year and then second question just around some of the frontline Ashish in a trial in outfit and Mel where you said that you were planning to meet with the regulators to use and market activity is a surrogate endpoint can you just talk a little more about the timing around that.
Speaker Change: How should we think about you are engaged with the FDA with the regulators on the timing for a margin of <unk> CR. Thank you.
Speaker Change: Great David Thanks for the question. So the first question is related to relapsed refractory <unk> and our timing relative to submission for our guidelines and does that will.
Speaker Change: Will that potentially happen before approval. So so a couple of steps here first we plan to publish the data I think we announced in our prepared remarks.
Speaker Change: <unk> or sorry submit the data for publication.
Speaker Change: In the second quarter, and we hope to get approval on.
The guidelines that is included in the guidelines sometime in the second quarter as well so that's.
Speaker Change: Certainly in advance of when the drug would be approved towards the end of this year.
Speaker Change: Which would of course provide us a very nice advantage.
Speaker Change: For being the first.
Speaker Change: To have guideline coverage for N P M. One and we believe to get the drug approved as well. So I think that's the timing for.
Speaker Change: <unk> in the end the strategy there.
Speaker Change: The second question I think there maybe Neil is going to clarify a little bit related to that.
Neil: The <unk> in a meeting with regulators go ahead, yes.
Speaker Change: So thanks for the question.
Speaker Change: Typically we don't discuss the details of our interactions with health authorities.
Speaker Change: I would just point out that our interest in M&A negative embody negativity is this is it.
Speaker Change: The biomarker is long standing and as I mentioned in the prepared remarks, we have been part of the.
Speaker Change: Consortium led by the NIH.
NIH, which also includes other industry partners as well as the FDA, but specifics we won't get into specifics of discussions with with health authorities.
Speaker Change: Excellent. Our next question will come from Justin <unk> with <unk>. Your line is now open.
Justin: Thanks for taking my question and congrats on the strong commercial start here, Michael maybe just a follow up on the prior question can you speak to your confidence of a broad recommendation by end CCN in the second quarter and then any color on payer discussions regarding reimbursement of patients with MTM mutations with support of abroad.
Speaker Change: <unk> guideline recommendation ahead of the formal potential FDA label expansion.
Speaker Change: Later this year.
Speaker Change: Yeah. Thanks, Jason appreciate it I think the a couple of questions. So the first around the confidence of broad coverage in MCT and I think I think are well see I mean, I think we feel very confident the data is supportive of approval, we think its practice changing and informative and important and I think from that perspective, we expect that it will be.
Speaker Change: Included in the guidelines and we will have.
Speaker Change: Very good access as a result of that.
Speaker Change: Second one is related to payer coverage, how that I guess that cup that follows on from there and whether we expect that to impact our ability to get the drug covered and maybe Steve if you want to make a comment about maybe even take it a step back in but we've had a good start I think in my prepared comments and Michael's comments on market access we're off to a good start per.
Speaker Change: Payer coverage is growing it's at 53% importantly claims are being paid really across the continuum.
Speaker Change: I think because we had such a early start with the payer team, calling it probably year and a half advance of approval calling on payers.
Speaker Change: The activity on NPM, one in the payer space will proceed that of the customer facing sales team motive I mean by that once we file we'll begin talking about NPM, one with payers, making sure. They are prepared just like they were for the <unk> launch I think in the interim Justin claims are being paid regardless of of vindication for all we can tell some of this is anecdotal but payers are not.
Speaker Change: Standing in the way of physicians, who need treatment, where they otherwise don't have one and they don't have one whether it's <unk> or M. P. M. One relapsed refractory <unk> patients in need and they see the need to to get patients on drug and are paying the claims. So we feel pretty good about where we are and getting to a better place as we near the launch of that indication.
Speaker Change: That makes sense to me and Steve if I could just ask a quick follow up could you just talk to in your opinion.
Speaker Change: <unk> being first.
Speaker Change: Competitive marketplace like the importance of having a first to market.
Speaker Change: Yes, I appreciate the question, Justin I think being first to market first mover advantages and something that syntax made up. This is just a function of the market and what we expect the kind of competitive immunity that we're building now it's about getting drug out is having the right customer facing field team the right trade support bright medical affairs alignment.
Speaker Change: Doing things right right out of the gates, which we are doing something we didnt I mentioned in my prepared comments, but I did I would amplify is time to first fill the ability to from prescription to getting patients on where we're seeing patients get on in a in a couple of days and anybody who takes longer its just because of payer, maybe giving us some challenges upfront, which ultimately get through so it's that type of.
Speaker Change: Great experience White glove experience that we're trying to provide for physicians and patients that will be very challenging for any follow on product if they make it to market to match, what we're doing the kind of experience that physicians will get in a year and possibly more of us being alone.
Speaker Change: Market might as well be 10 years that that's the kind of muscle memory that accounts gain and thats what our goal is.
Speaker Change: Thanks for taking my questions and congrats again.
Speaker Change: Thank you Joseph.
George Farmer: Our next question comes from George Farmer with Scotiabank. Your line is now open.
George Farmer: Hi, Good morning, Thanks for taking my questions a couple from me.
George Farmer: Regarding the strategy for.
George Farmer: Obtaining a compendium listing in the NPM untapped population can you point to other AML drugs in this space that have secured compendium listing based on similar data set than what you have with what you have today.
George Farmer: And then as a.
George Farmer: A follow up regarding your strategy in the intensive chemo fit patient population.
George Farmer: This data that debt.
George Farmer: Could come in the second half in combination can you talk about what you think the optimal doses in combination with IC.
George Farmer: Thanks.
George Farmer: Great. Thanks George.
Speaker Change: So in terms of your question. Your first question was strategy for listing for MTM, one and can we point to some other other products that were listed and potentially in a similar capacity I think that was the question I, usually maybe you want to take that yeah. Thanks George.
Speaker Change: Couple of examples in the states. So I think the first example is sort of asking is on phase II data set theory granted an <unk> guideline.
Speaker Change: Listing for foot three.
Speaker Change: Treatment.
Speaker Change: In frontline and then.
Speaker Change: I think more recently <unk> in combination with <unk> off of Phase II dataset.
Speaker Change: Hello, I am childless granted continue listening as well. So I think there is plenty of examples I mean, even our therapy for KN <unk> gotten.
Speaker Change: So I think we.
Speaker Change: In highlight thanks.
Speaker Change: When we get there.
Speaker Change: Alright, and maybe thank you and then the second question.
Speaker Change: Sorry, George I think the second question relates to a combo with chemotherapy and the dose and have the selection is gonna work, maybe I'll turn it over to Neil Yeah look thanks for the question.
Speaker Change: As mentioned, we'll present data later in the year.
Speaker Change: We are just just to sort of summarize where.
Speaker Change: We are at this point in terms of all of the combinations that we spend that we've been testing revenue might have been the <unk>.
Speaker Change: <unk> has been actually well.
Speaker Change: Tolerated at Boe.
Speaker Change: <unk> doses tested both.
Speaker Change: <unk> $160 so.
Speaker Change: We have choices right it could be either dose.
Speaker Change: The data will indicate to us.
Speaker Change: Which dose it could be currently we're combining a full dose.
Speaker Change: That's what we're doing.
Speaker Change: Currently and as I said, we'll report more later on there.
Speaker Change: Excellent thanks very much.
Speaker Change: Thank you George.
Speaker Change: Our next question will come from Salim Syed with Mizuho. Your line is now open.
Speaker Change: Hi, This is Eric on for Celine Thanks for taking my question and congrats on the.
Speaker Change: The nice ramp for <unk>.
Speaker Change: So I wanted to.
Speaker Change: Try to get some color on what you think of.
Speaker Change: The.
Speaker Change: Timing and that.
Speaker Change: Total addressable size of ex U S for <unk>.
Speaker Change: What are your thoughts there on.
Speaker Change: What the gating factors are.
Speaker Change: That we would need to see for you to.
Speaker Change: To go for ex U S regulatory submissions and what that might look like if it's a collaboration license something like that thank you.
Speaker Change: Great. Thanks, Thanks, Eric for the question. So first I think you had asked about timing for ex U S.
Speaker Change: And I think the.
Speaker Change: We're we haven't announced all of our plans regulatory and development wise for ex U S. I think they do come together, rather nicely around our frontline strategy in getting the drug approved ex U S will be tied to not only the frontline trials, but there may be other opportunities as well. So we have we're.
Speaker Change: We haven't been.
Speaker Change: Specific about the timing I think the addressable market is probably about half of the U S.
Speaker Change: Ex U S. It's a very very.
Speaker Change: Appreciable market opportunity for us So we'll look to address it as soon as possible and as I mentioned in my in my earlier remarks looking for part looking for a partner to help us certainly with revenue for us outside the U S that will come in time as well as we look to commercialize and develop more extensively.
Speaker Change: As outlined so I think that's a that's a ongoing development with with our store as you know for Nick Tivo. We do have we do have a partner in insight and they will commercialize and develop the knick timber outside the United States. So over the next couple of years I think youll see a lot more development and.
Speaker Change: And plans to move globally.
Speaker Change: Alright helpful. Thank you.
Speaker Change: Thanks.
Speaker Change: Our final question will come from the line of Jason <unk> with Bank of America. Your line is now open.
Jason <unk>: Good morning, Congratulations on the launch progress and thanks for taking our questions.
Speaker Change: Two if I may somewhat of a follow up but regarding the Hogan led trial in the unfit population, there's something in the protocol.
Speaker Change: A potential path to accelerated approval and what was the timeline look like for that.
Speaker Change: And then maybe from a broader strategic perspective, how are you prioritizing first line fit versus unfit, what's the more important opportunity for revenue, especially considering the evolving competitive landscape.
Speaker Change: Great.
Speaker Change: Jason. Thank you so much for the questions first one relates to the whole von trial.
Speaker Change: An accelerated approval and timeline and I'm going to turn that over to Neil.
Speaker Change: So thanks for the question as I mentioned in the prepared remarks, we are positioned and we will continue to position yourself.
Speaker Change: To be able to avail of any opportunity.
Speaker Change: For accelerated approval based on intermediate endpoints, however, where we are.
Speaker Change: It does not our not our practice to discuss the details of our discussions with health authorities.
Speaker Change: Alright, and maybe.
Speaker Change: Maybe first line fit versus onset prioritization was the second question I could take that one look I think we're the unfit populations and as a combination trial with hold on I think that that addresses the highest unmet need in the frontline population and we will be first to initiate a trial there.
Speaker Change: Also have the only data set in that population to distinguish ourselves. So I think that is a very important first trial to initiate I think we will have other trials as Neil pointed out unique fit population.
Speaker Change: Initiated before the end of this year that will help to kind of drive.
Speaker Change: Our strategy is set so I think in 2025, we will take a quantum leap forward in initiating trials that will address all the important populations both fit and unfit.
Speaker Change: But the first trial to initiate will be the hold on trial, which again is a global global registration trial in that population. So it's again, it's around unmet need and it's around the opportunities in front of us and we plan to be first to capitalize on all of these market opportunities.
Speaker Change: Great. Thanks, Mike I appreciate the color.
Speaker Change: Thank you.
Speaker Change: This concludes the question and answer session I will now turn the floor over to Mr. Michael That's correct for any additional comments or closing remarks.
Speaker Change: Great. Thank you operator, and thank you all we really appreciate you all tuning in today to discuss our recent progress and the exciting milestones ahead, we look forward to seeing many of you at the Cowen Jefferies and Barclays conferences, this month and with that have a fantastic day.
Speaker Change: Yeah.
Speaker Change: Okay.
Speaker Change: [music].
Speaker Change: Yeah.
Speaker Change: Sure.
Yes.
Speaker Change: Yes.
Speaker Change: Yes.
Yeah.
Speaker Change: Yes.
Speaker Change: Yes.